Differences in the Reimbursement Pathway for Ultraorphan Therapies in England and Ireland
Author(s)
Pratik K. Bal, MPH, BDS1, Jennifer Knight, PhD2, Annie Barnes, PhD2, Carl Prescott, MPH2, Brenda Dooley, MSc1.
1AXIS - The Reimbursement Experts, Dublin, Ireland, 2AXIS - The Reimbursement Experts, London, United Kingdom.
1AXIS - The Reimbursement Experts, Dublin, Ireland, 2AXIS - The Reimbursement Experts, London, United Kingdom.
OBJECTIVES: In Ireland and England, Health Technology Assessments (HTAs) are conducted by the National Centre for Pharmacoeconomics (NCPE) and the National Institute for Health and Care Excellence (NICE), respectively. This study aimed to compare the timelines and outcomes of HTA processes for ultra-orphan therapies across both agencies and assess their impact on patient access.
METHODS: We examined HTA submissions for ultra-orphan therapies made between 2022 and 2025. NICE submissions were identified through the Highly Specialised Technology (HST) program. Corresponding NCPE assessments, including rapid reviews and full HTAs, And the outcomes and timelines of the appraisals were extracted were extracted from the NCPE website. Regulatory approval dates were sourced from the websites of European Medicines Agency (EMA), Orphanet and the Medicines and Healthcare products Regulatory Agency (MHRA). Further comparison was made between ultra-orphan and orphan therapies evaluated by the NCPE.
RESULTS: Twenty-one ultra-orphan therapies were submitted to NICE via the HST pathway, of which twelve were also assessed by the NCPE. The average time to reimbursement was 528 days for NICE and 1,093 days for the NCPE. Of the remaining nine therapies, none had entered the NCPE process during the study period. To assess whether this delay was specific to ultra-orphan therapies, NCPE timelines for orphan therapies were compared. Among 54 orphan therapy submissions to NCPE, 21 received positive HSE-reimbursement, with an average processing time of 661 days-432 days shorter than that for ultra-orphan therapies.
CONCLUSIONS: The findings show a significant difference in the time to reimbursement of ultra-orphan disease therapies between NICE and NCPE. These delays may result in reduced or delayed access to potentially life-changing treatments. Such prolonged timelines may lead to worsened health outcomes and increased disparities. Further research is needed to understand rationale for the delay and improve timelines for patients.
METHODS: We examined HTA submissions for ultra-orphan therapies made between 2022 and 2025. NICE submissions were identified through the Highly Specialised Technology (HST) program. Corresponding NCPE assessments, including rapid reviews and full HTAs, And the outcomes and timelines of the appraisals were extracted were extracted from the NCPE website. Regulatory approval dates were sourced from the websites of European Medicines Agency (EMA), Orphanet and the Medicines and Healthcare products Regulatory Agency (MHRA). Further comparison was made between ultra-orphan and orphan therapies evaluated by the NCPE.
RESULTS: Twenty-one ultra-orphan therapies were submitted to NICE via the HST pathway, of which twelve were also assessed by the NCPE. The average time to reimbursement was 528 days for NICE and 1,093 days for the NCPE. Of the remaining nine therapies, none had entered the NCPE process during the study period. To assess whether this delay was specific to ultra-orphan therapies, NCPE timelines for orphan therapies were compared. Among 54 orphan therapy submissions to NCPE, 21 received positive HSE-reimbursement, with an average processing time of 661 days-432 days shorter than that for ultra-orphan therapies.
CONCLUSIONS: The findings show a significant difference in the time to reimbursement of ultra-orphan disease therapies between NICE and NCPE. These delays may result in reduced or delayed access to potentially life-changing treatments. Such prolonged timelines may lead to worsened health outcomes and increased disparities. Further research is needed to understand rationale for the delay and improve timelines for patients.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA110
Topic
Health Technology Assessment, Organizational Practices, Real World Data & Information Systems
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases